Latest news

• Bluebird bio’s sickle cell gene therapy shows early-study benefit

  Closely follows approval of beta thalassaemia therapy Zynteglo. Bluebird bio’s gene therapy for sickle cell disease (SCD), LentiGlobin, has demonstrated significant benefit in a small group of patients treated in ... The two patients both had severe

• BMS, Acceleron Pharma’s Reblozyl to be reviewed by FDA expert panel

  The announcement closely follows news that the FDA approved  Reblozyl (luspatercept) for the treatment of anaemia caused by the rare blood disorder beta thalassaemia,  who regularly require red blood cell transfusions. ... The approval of Reblozyl in

• FDA backs GBT’s sickle cell drug Oxbryta

  One potential gene therapy – Bluebird Bio’s  Zynteglo – was given conditional approval in Europe for another blood disease called beta thalassaemia in June and is in late-stage development for SCD.

• Vertex reveals promising data from first CRISPR-treated patients

  The two patients have severe haemoglobinopathies – including transfusion-dependant beta thalassaemia (TDT) and severe sickle cell disease (SCD). ... beta thalassemia,” said Jeffrey Leiden (pictured left), president and chief executive officer of

• Novartis’ Mayzent and Roche’s Polivy lead CHMP recommendations

  Deferasirox Accord (deferasirox) for the treatment of chronic iron overload due to blood transfusions in patients with beta thalassaemia and other anaemia types.